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Nucleic Acids Research|April 29, 2025
Donor insertion into CX3CR1 allows epigenetic modulation of a constitutive promoter on hematopoietic stem cells and its activation upon myeloid differentiationIris Ramos-Hernández, Carla Fuster-García, Araceli Aguilar-González, et al.
Nature Methods|September 23, 2024
Effective genome editing with an enhanced ISDra2 TnpB system and deep learning-predicted ωRNAsKim Fabiano Marquart, Nicolas Mathis, Amina Mollaysa, et al.
Molecular Therapy. Methods & Clinical Development|February 12, 2021
Automated generation of gene-edited CAR T cells at clinical scaleJamal Alzubi, Dominik Lock, Manuel Rhiel, et al.
Cellular and Molecular Life Sciences : CMLS|September 22, 2022
CAPRIN1P512L causes aberrant protein aggregation and associates with early-onset ataxiaAndrea Delle Vedove, Janani Natarajan, Ginevra Zanni, et al.
Orphanet Journal of Rare Diseases|May 15, 2025
TREAT: systematic and inclusive selection process of genes for genomic newborn screening as part of the Screen4Care projectChristina Saier, Stefaan Sansen, Joanne Berghout, et al.
The Journal of Allergy and Clinical Immunology|December 17, 2009
ORAI1 deficiency and lack of store-operated Ca2+ entry cause immunodeficiency, myopathy, and ectodermal dysplasiaChristie-Ann McCarl, Capucine Picard, Sara Khalil, et al.
Der Nervenarzt|May 13, 2020
[Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM)]Andreas Ziegler, Ekkehard Wilichowski, Ulrike Schara, et al.
Journal of Neurology|April 28, 2023
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysisEugenio Mercuri, Andrés Nascimento Osorio, Francesco Muntoni, et al.
Orphanet Journal of Rare Diseases|May 9, 2025
The most bothersome symptoms in neuromuscular diseases: the ERN EURO NMD SurveyMichelangelo Mancuso, Alessandro Colitta, Manuela Lavorato, et al.
Cell Reports. Medicine|December 5, 2024
Modulation of TCR stimulation and pifithrin-α improve the genomic safety profile of CRISPR-engineered human T cellsLaurenz T Ursch, Jule S Müschen, Julia Ritter, et al.
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